This section provides an introduction to the
subject of benefit:risk analysis and also contains
a list of definitions of pharmacoepidemiologic and biostatistical
terms that are applicable to benefit:risk assessments.
The process of benefit:risk analysis is a continuous evaluation of the benefits and risks of
drugs and biologic products that is started by pharmaceutical companies
and the US Food and Drug Administration (FDA) during the new drug
development and approval process and continues through the life cycle
of the product. Benefit:risk analyses are done
every day by medical practitioners (e.g., doctors, nurses, and pharmacists),
health planners, epidemiologists, and patients.6131 There
are a host of considerations the FDA and pharmaceutical companies
weigh in seeking approval for medications, and these considerations
also influence how medical practitioners select the most appropriate
treatments for their patients.
The benefit:risk analysis
of drugs and biologic products involves the collection and assessment
of a multitude of data on the safety and efficacy of these interventions.
A range of sources for such data is available to the medical researcher.6132 These
sources include clinical trials, observational trials, insurance claims,
electronic medical records, registries, surveys, surveillance databases,
and population longitudinal cohorts. Randomized, controlled clinical
trials are considered the gold standard for establishing the benefit:risk profile of a product prior to it being marketed.
Additional data, primarily from postmarketing safety surveillance
and from other sources, contribute to the ongoing benefit:risk analysis of marketed products. Each of these data sources has strengths
and potential limitations.
Pharmacoepidemiology includes the systematic
study of drug effects in discrete (nonrandomized) populations. Pharmacoepidemiology
can also consider randomized analyses such as clinical trials.6133 Results
of pharmacoepidemiologic studies are used to estimate the expected
rates of events in the course of an illness or an intervention. A
range of study designs and observational research methods form a key
part of the overall benefit:risk analysis of drugs
and biologic products.
Health outcomes research encompasses both health
economics research and pharmacoeconomics, disciplines that are important
to benefit:risk analysis. Health outcomes research
evaluates the clinical, economic, and humanistic consequences of specific
health care interventions or treatment plans, such as patient-reported
outcomes and health economics. Health economics has been defined as
"the description and analysis of the costs of drug therapy to healthcare
systems and society."7198
Benefit:risk analyses occur
daily and their outcomes affect all facets of our lives. These analyses
are not restricted to health care alone; benefit:risk decisions involve business, academia, commerce, government, and
each one of us as individuals. The Benefit:Risk Case Examples section describes 3 cases that were well-publicized: the use of
mammography, the use of nuclear power, and the development of rotavirus
vaccines. They help to demonstrate how benefit:risk analyses are utilized in the real world.